Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults (D1AMOND)

A Multicenter, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Maintenance of Efficacy of Ecopipam in Children, Adolescents and Adults With Tourette's Disorder

This Phase 3 multicenter study evaluates the maintenance of efficacy, safety and tolerability of ecopipam tablets in children, adolescents and adults in the treatment of Tourette's Disorder (TD). The study includes an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period.

Study Overview

• Status: Completed
• Conditions: Tourette Disorder
• Intervention / Treatment: Drug: Ecopipam Hydrochloride

Detailed Description

Following a 28-day Screening period and Baseline visit, eligible subjects will be enrolled into the open-label Stabilization period comprised of a 4-week Titration phase to achieve a target steady-state dose of 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) followed by an 8-week open-label Maintenance phase. Responders to ecopipam will be randomized to ecopipam 1.8 mg/kg/day (2 mg/kg/day ecopipam HCl) or placebo in a 1:1 fashion and enter the double-blind Randomized-Withdrawal (R/WD) period at Week 12. During the 12-week R/WD period, any subject meeting Relapse criteria will be withdrawn from blinded study medication and complete end of study assessments including safety follow up visits.

• Study Type: Interventional
• Enrollment (Actual): 216
• Phase: Phase 3

Contacts and Locations

Study Locations

• Bulgaria
  • Sofia, Bulgaria, 1680
    • Kalimat Medical Center_Sofia
  • Varna, Bulgaria, 9020
    • DCC Mladost-M Varna
  • Bulgaria
    • Plovdiv, Bulgaria, Bulgaria, 4004
      • Center Spectar-Plovdiv
  • Sofia-Grad
    • Sofia, Sofia-Grad, Bulgaria, 1360
      • ASMP-IP- d-r Kayryakova
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2N 4Z6
      • University of Calgary
  • Ontario
    • Ajax, Ontario, Canada, L1Z 0M1
      • The Kids Clinic Inc
• Denmark
  • Herlev, Denmark, 02730
    • Borne og Ungeafdelingen
• France
  • Grenoble, France, 38000
    • CHU GRENOBLE ALPES - Hôpital COUPLE ENFANT
  • Paris, France, 75019
    • Hôpital Fondation Rothschild
  • Strasbourg, France, 67098
    • CHU Strasbourg-Hopital de Hautepierre
• Germany
  • Mannheim, Germany, 68159
    • Zentralinstitut fuer Seelische Gesundheit, Mannheim
  • Bavaria
    • Munich, Bavaria, Germany, 80336
      • Psychiatric Clinic of Ludwig Maximilians Universitaet Muenchen
  • Lower Saxony
    • Hanover, Lower Saxony, Germany, 30625
      • Department of Psychiatry, Socialpsychiatry and Psychotherapy, Hannover Medical School
• Hungary
  • Budapest, Hungary, 1021
    • Vadaskert Gyermek- es Ifjusagpszichiatriai Korhaz es Szakambulancia
  • Budapest, Hungary, 1146
    • Bethesda Childrens Hospital(Magyarországi Református Egyház Bethesda Gyermekkórháza)
  • Csongrád megye
    • Szeged, Csongrád megye, Hungary, 6725
      • Szegedi Tudomanyegyetem Altalanos Orvostudomanyi Kar Gyermek
• Italy
  • Catania, Italy, 95123
    • Azienda Ospedaliera Vittorio Emanuele Policlinico // Azienda Ospedaliera Policlinico San Marco
  • Genova, Italy, 16148
    • Ospedale Pediatrico Istituto Giannina Gaslini di Genova
  • LC
    • Bosisio Parini, LC, Italy, 23842
      • Istituto Di Ricovero E Cura A Carattere Scientifico IRCCS Eugenio Medea
  • Milano
    • Milan, Milano, Italy, 20133
      • IRCCS Istituto Neurologico Carlo Besta
  • Napoli
    • Napoli, Napoli, Italy, 80131
      • Università degli Studi di Napoli Federico II
  • RM
    • Roma, RM, Italy, 00165
      • Ospedale Pediatrico Bambino Gesu
• Poland
  • Krakow, Poland, 30-363
    • Centrum Medyczne Plejady
  • Poland
    • Poznan, Poland, Poland, 61-731
      • Clinical Research Center Sp. z o.o. Medic-R sp.k.
  • Pomeranian Voivodeship
    • Gdansk, Pomeranian Voivodeship, Poland, 80-542
      • Gdanskie Centrum Zdrowia Sp z o.o.
  • Silesian Voivodeship
    • Katowice, Silesian Voivodeship, Poland, 42-123
      • Wielospecjalistyczna Poradnia Lekarska Synapsis
  • Woj. Malopolskie
    • Krakow, Woj. Malopolskie, Poland, 31-503
      • Wojewdzki Specjalistyczny Szpital Dziecicy im. sw. Ludwika w Krakowie
• Romania
  • Dorobanți, Romania, 41914
    • Spitalul Clinic de Psihiatrie Prof Dr Al. Obregia Bucureti
• Serbia
  • Belgrade, Serbia, 11000
    • Clinic of Neurology and Psychiatry for Children and Adolescents
  • Belgrad
    • Belgrade, Belgrad, Serbia, 11120
      • Institute of Mental Health
  • Serbia
    • Niš, Serbia, Serbia, 18000
      • Clinical Centre Nis Center of Mental Health
    • Novi Sad, Serbia, Serbia, 21000
      • Clinical Center Vojvodina
• Spain
  • Barcelona, Spain, 8036
    • Hospital Clínic de Barcelona
  • Seville, Spain, 41009
    • Hospital Universitario Virgen Macarena Unidad de Investigacion Neurologia
  • Madrid
    • Alcorcón, Madrid, Spain, 28922
      • Hospital Universitario Fundacion Alcorcon HUFA
    • Madrid, Madrid, Spain, 28041
      • Hospital Universitario 12 de octubre
    • Madrid, Madrid, Spain, 28007
      • Hospital General Universitario Gregorio Marañon
  • Principality of Asturias
    • Oviedo, Principality of Asturias, Spain, 33011
      • Hospital Universitario Central de Asturias
  • Sevilla
    • Seville, Sevilla, Spain, 41013
      • Hospital Universitario Virgen del Rocío, C/Antonio Maura Montaner, s/n, edificio IBiS
• United States
  • Alabama
    • Dothan, Alabama, United States, 36303
      • Harmonex Neuroscience Research
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Anaheim, California, United States, 92805
      • Advanced Research Center
    • Bellflower, California, United States, 90706
      • CenExel CIT-IE
    • Glendale, California, United States, 91203
      • Cortica Site Network
    • Irvine, California, United States, 92604
      • Amnova Clinical Research
    • San Rafael, California, United States, 94903
      • Cortica Site Network - San Rafael
    • Santa Ana, California, United States, 92705
      • Syrentis Clinical Research
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale School of Medicine
  • Florida
    • Gainesville, Florida, United States, 32608
      • University of Florida
    • Gulf Breeze, Florida, United States, 32561
      • NW FL Clinical Research Group, LLC
    • Hialeah, Florida, United States, 33013-3834
      • Research in Miami Inc
    • Jensen Beach, Florida, United States, 34957
      • Emcrown Clinical Research
    • Miami, Florida, United States, 33136
      • University of Miami Miller School of Medicine
    • Miami, Florida, United States, 33173
      • Florida International Research Center
    • Miami, Florida, United States, 33175
      • Care Research Center
    • Orange City, Florida, United States, 32763
      • Medical Research Group of Central Florida
    • Orlando, Florida, United States, 32803
      • APG Research LLC
    • St. Petersburg, Florida, United States, 33701
      • University of South Florida
    • Tampa, Florida, United States, 33609-4181
      • Pediatric Epilepsy and Neurology Specialists
    • Tampa, Florida, United States, 33617
      • Jedidiah Clinical Research
    • Winter Park, Florida, United States, 32789
      • Pediatric Neurology, PA
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Rare Disease Research, LLC
    • Atlanta, Georgia, United States, 30338
      • Atlanta Behavioral Research, LLC.
    • Atlanta, Georgia, United States, 30318
      • Advanced Discovery Research, Llc
  • Illinois
    • Chicago, Illinois, United States, 60637-1447
      • The University of Chicago Hospitals
    • Chicago, Illinois, United States, 60611
      • Lurie Children Hospital of Chicago
    • Chicago, Illinois, United States, 60612
      • 1725 W. Harrison St., Suite 755
  • Indiana
    • Indianapolis, Indiana, United States, 46256
      • Josephson-Wallack-Munshower Neurology
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
    • Worcester, Massachusetts, United States, 01655
      • UMass Chan Medical School
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Michigan Clinical Research Institute PC
    • Bloomfield Hills, Michigan, United States, 48302-1952
      • NeuroBehavioral Medicine Group
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • The Childrens Mercy Hospital
    • Saint Charles, Missouri, United States, 63304
      • Midwest Research GRoup
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Alivation Research
  • New York
    • New York, New York, United States, 10029-6504
      • Mount Sinai School of Medicine
    • New York, New York, United States, 10016-5815
      • NYU Child Study Center
    • Rochester, New York, United States, 14642
      • University of Rochester
    • Stony Brook, New York, United States, 11794
      • Stony Brook University Medical Center
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157-0001
      • Wake Forest Baptist Medical Center - PPDS
  • Ohio
    • Cincinnati, Ohio, United States, 45229-3026
      • Cincinnati Childrens Hospital Medical Center
    • Middleburg Heights, Ohio, United States, 44130
      • North Star Medical Research, LLC
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Suburban Research Associates
  • South Carolina
    • Charleston, South Carolina, United States, 29414
      • Coastal Pediatric Research
  • Tennessee
    • Nashville, Tennessee, United States, 37203-6502
      • Access Clinical Trials, Inc.
    • Nashville, Tennessee, United States, 37232-7610
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine
    • Houston, Texas, United States, 77030
      • The University of Texas Health Science Center at Houston
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital (TCH)
    • Richmond, Texas, United States, 77407
      • Perceptive Pharma Research (PPR)
    • San Antonio, Texas, United States, 78249-3539
      • Road Runner Research Ltd.
  • Utah
    • Draper, Utah, United States, 84020
      • Cedar Clinical Research
  • Washington
    • Everett, Washington, United States, 98201
      • Core Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• ≥ 6 years of age
• ≥ 18 kg (~ 40 lbs.)
• TD diagnosis and both motor and vocal tics that cause impairment with normal routines
• Minimum score of 20 on the YGTSS-R Total Tic Score
• May not be taking any medications used to treat motor or vocal tics for at least 14 days prior to Baseline.
• Effective contraception during the study and 30 days after last study dose for sexually active subjects

Exclusion Criteria:

• Previous exposure to ecopipam
• Certain mood or psychiatric disorders (i.e., dementia, bipolar disorder, schizophrenia, major depressive disorder)
• Unstable medical illness or clinically significant lab abnormalities
• Risk of suicide
• Pregnant or lactating women
• Moderate to severe renal insufficiency
• Hepatic insufficiency
• Positive urine drug screen
• Unstable doses for drugs to treat anxiety, depression, Attention Deficit Hyperactivity Disorder
• Certain medications that would lead to drug interactions
• Recent behavioral therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl); Ecopipam 11.2, 22.4, 33.6, 44.8, 67.2 and 89.6 mg tablets (containing 12.5, 25, 37.5, 50, 75 and 100 mg ecopipam HCl, respectively); 1.8 mg/kg/day ecopipam (2 mg/kg/day ecopipam HCl) target dose; oral administration daily in evenings.	Drug: Ecopipam Hydrochloride; Selective dopamine D1 and D5 receptor antagonist
Placebo Comparator: Placebo during R/WD Phase; Matching Placebo tablets during R/WD period taken orally in the evening.	Drug: Ecopipam Hydrochloride; Selective dopamine D1 and D5 receptor antagonist

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time From Randomization to Relapse in Participants Greater Than and Equal to (>=) 6 and Less Than (<) 18 Years During the Double-Blind R/WD	Time to relapse defined as a loss of >=50 percent (%) of the improvement experienced on the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of Tourette's Disorder (TD), or requirement of hospitalization for worsening symptoms of TD in participants between the ages of >= 6 and <18 years for ecopipam compared to those receiving placebo during the double-blind, R/WD period. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in participants with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.	From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time From Randomization to Relapse in All Participants During the Double-Blind R/WD Period	Time From Randomization (Week 12) to relapse defined as a loss of >= 50% of the improvement experienced on the YGTSS-TTS from Baseline to the last visit in the Open-Label Stabilization Period (Week 12), or initiation of additional medications to treat symptoms of TD, or requirement of hospitalization for worsening symptoms of TD in all participants during the Double-Blind R/WD period for ecopipam compared to placebo. The YGTSS was a clinician-completed rating scale used to quantify overall tic severity in children and adults with TD as well as specific subdomains of tic number, frequency, intensity, complexity and interference. Each of these subdomains was scored, on a 0 to 5 scale, separately for motor and vocal tics and then summed across both motor and vocal tics to yield a total tic score ranging from 0 to 50. Higher scores represented more severe symptoms.	From randomization at Week 12 through the end of the double-blind R/WD period (up to Week 24)

Collaborators and Investigators

• Sponsor: Emalex Biosciences Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 31, 2023
• Primary Completion (Actual): January 13, 2025
• Study Completion (Actual): February 4, 2025

Study Registration Dates

• First Submitted: November 7, 2022
• First Submitted That Met QC Criteria: November 7, 2022
• First Posted (Actual): November 14, 2022

Study Record Updates

• Last Update Posted (Estimated): November 25, 2025
• Last Update Submitted That Met QC Criteria: November 12, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Genetic Diseases, Inborn; Neurodegenerative Diseases; Neurodevelopmental Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Tic Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Tourette Syndrome; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Neurotransmitter Agents; Tranquilizing Agents; Psychotropic Drugs; Dopamine Agents; Antipsychotic Agents; Dopamine Antagonists; Ecopipam
• Other Study ID Numbers: EBS-101-TD-301; 2023-503494-38-00 (Ctis); 2022-001961-11 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No